Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1-Reference-Cited by-同舟云学术

Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1

Published:2019-03-16 Issue:5 Volume:36 Page:1164-1176
ISSN:0741-238X
Container-title:Advances in Therapy
language:en
Short-container-title:Adv Ther

Author:

Dabbous Omar,Maru Benit,Jansen Jeroen P.,Lorenzi Maria,Cloutier Martin,Guérin Annie,Pivneva Irina,Wu Eric Q.,Arjunji Ramesh,Feltner Douglas,Sproule Douglas M.

Funder

AveXis

Publisher

Springer Science and Business Media LLC

Link

http://link.springer.com/content/pdf/10.1007/s12325-019-00923-8.pdf

Reference26 articles.

1. D’Amico A, Mercuri E, Tiziano FD, Bertini E. Spinal muscular atrophy. Orphanet J Rare Dis. 2011;6:71.

2. Ogino S, Leonard DGB, Rennert H, Wilson RB. Spinal muscular atrophy genetic testing experience at an academic medical center. J Mol Diagn. 2002;4(1):53–8.

3. Parente V, Corti S. Advances in spinal muscular atrophy therapeutics. Ther Adv Neurol Disord. 2018;11:1756285618754501.

4. Verhaart IEC, Robertson A, Wilson IJ, Aartsma-Rus A, Cameron S, Jones CC, et al. Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy—a literature review. Orphanet J Rare Dis. 2017;12(1):124.

5. Lefebvre S, Burglen L, Reboullet S, Clermont O, Burlet P, Viollet L, et al. Identification and characterization of a spinal muscular atrophy-determining gene. Cell. 1995;80(1):155–65.

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